A new research project on rare cystic fibrosis (CF) mutations has received $600,000 in funding over three years from Génome Québec and Cystic Fibrosis Canada. Led by Larry Lands, MD, PhD, and Dr. Jacques-P. Tremblay (Laval University), the study focuses on developing a genetic therapy for CF patients with rare mutations that do not respond to current treatments. The project, “Leave No One Behind: development of a genetic therapy for people living with cystic fibrosis who do not respond to available modulators,” proposes using prime editing to correct CFTR gene mutations in airway cells via injection or inhalation of lipid nanoparticles. A cell model with a common Canadian CFTR mutation will be created to test therapy effectiveness. The multidisciplinary team includes experts from McGill, SickKids, Concordia, and Laval including Darcy Wagner, PhD and Jun Ding, PhD, both of McGill. The research could pave the way for clinical trials, offering new treatment options for those without effective therapies.
Dr. Lands was recently awarded the King Charles III Coronation Medal for his contributions to CF research.
Congratulations Dr. Lands!
Read more:
New funding for cystic fibrosis research, The Institute (Montreal Children’s Hospital) March 17, 2025
Leave No One Behind: development of a genetic therapy for people living with Cystic Fibrosis who do not respond to available modulators. Génome Québec
Génome Québec and Cystic Fibrosis Canada invest $1.2 million in research tackling rare, orphan CFTR mutations. Cystic Fibrosis Canada
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